Autolus Therapeutics (AUTL) FY Conference June 11, 2025 01:20 PM ET Speaker0 Good afternoon, everyone, and thank you for joining. My name is Rajan Sharma, European pharma and biotech analyst here at Goldman Sachs. I'm very pleased that we have Rob Dolsky, Chief Financial Officer at Autolus. Rob, you so much for joining us. Speaker1 Great to be here. Speaker0 Maybe kind of just to start for people who may be less familiar with the Autolus story, could you just kind of give us an overview of the company, key ...

Summary of 4D Molecular Therapeutics (FDMT) FY Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (FDMT) - **Focus**: Gene therapy with a focus on ophthalmology and pulmonology, specifically targeting conditions like wet age-related macular degeneration (AMD), diabetic macular edema (DME), and cystic fibrosis. Key Points Industry and Market Opportunities - **Gene Therapy Potential**: The company believes it has developed best-in-class vectors for various delivery methods, which opens up large sustainable markets in gene therapy [1][2] - **Market Size**: The potential market opportunities are significant, with estimates of 4,150 million for wet AMD and DME, and 4,710 million for cystic fibrosis lung disease [1] Clinical Development and Trials - **Phase III Trials**: The focus for the next twelve months is on executing Phase III trials for FORWARD I and FORWARD II in wet AMD and initial dose data for cystic fibrosis [2] - **Physician Feedback**: Positive feedback from physicians regarding the durability of treatments and reduced injection burden, with expectations of an 80-90% reduction compared to existing therapies [6][7] - **Safety Profile**: The company reports a strong safety profile with no inflammation observed in DME trials, which is critical for large markets like wet AMD and DME [9][10] Commercial Strategy - **Pricing Flexibility**: The cost of goods for treatments is around $500, allowing for competitive pricing strategies that can benefit patients and shareholders [26] - **Durability Demand**: Surveys indicate that durability and extended treatment duration are the top desires for both patients and physicians, positioning gene therapy as a leading option [22] Regulatory and Financial Position - **Regulatory Engagement**: Ongoing discussions with the FDA regarding potential accelerated approval pathways for cystic fibrosis treatments, especially for patients with no available therapies [41][43] - **Financial Health**: The company ended Q1 with $458 million in cash, primarily allocated to fund Phase III trials for wet AMD, with a runway extending into 2028 [51] Future Directions - **Partnership Opportunities**: The company is open to partnerships outside the U.S. for its products, particularly in the lung franchise, while retaining U.S. rights for ophthalmology [48][49] - **Platform Development**: The gene therapy platform has shown broad utility, with six open INDs and a focus on developing a portfolio of products efficiently [45][54] Additional Insights - **Market Differentiation**: The company emphasizes that not all gene therapies are the same, highlighting its unique approach to local application and cost management, which differentiates it from other programs in the field [56] This summary encapsulates the key insights and strategic directions discussed during the conference call, providing a comprehensive overview of 4D Molecular Therapeutics' current status and future outlook in the gene therapy market.

Prelude Therapeutics (PRLD) FY Conference June 11, 2025 09:20 AM ET Speaker0 Are necessary for a successful SMARK A2 directed agent? Obviously, sounds like the greater over inhibitor is one of the But what are some of Speaker1 the key properties you think are necessary for this kind of agent to be successful? Yes. So we know from our preclinical models that if you hit both four and two that you have quite a bit of toxicity. And so the degradation of II allows and selectivity and potency for II actually allo ...

-- PCRX-201 demonstrated sustained clinical efficacy with improvements in pain, stiffness, and function for up to three years – BRISBANE, Calif., June 11, 2025 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (NASDAQ: PCRX), the industry leader in the delivery of innovative, non-opioid pain therapies to transform the lives of patients, today announced new long-term follow-up data from its Phase 1 clinical trial evaluating PCRX-201 (enekinragene inzadenovec), a novel gene therapy candidate for osteoarthritis of ...

SYDNEY, June 11, 2025 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a clinical-stage biotechnology company pioneering next-generation oncology therapeutics, today announced the publication of transformative preclinical research in the journal Molecular Cancer Therapeutics, a journal of the American Association for Cancer Research that underscores the powerful potential of its lead asset, paxalisib, in reshaping the treatment landscape for triple-negative breast cancer (TNBC), one of the most ag ...

~ Proven biotech executive to lead commercialization of AMT-130 in Huntington’s disease ~ LEXINGTON, Mass. and AMSTERDAM, June 11, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Kylie O’Keefe as Chief Customer and Strategy Officer, effective June 6, 2025. In this role, Ms. O’Keefe will lead the development and execution of uniQure’s global commercialization stra ...

WILMETTE, Ill. and HOUSTON, Texas, June 11, 2025 (GLOBE NEWSWIRE) -- Monopar Therapeutics Inc. ("Monopar," the "Company," "us" and "our") (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with unmet medical needs, in collaboration with Excel Diagnostics and Nuclear Oncology Center ("EDNOC"), a premier diagnostic medical imaging and therapeutic nuclear medicine center, today announced that the physician-sponsored Expanded Access Program ("EAP" ...

-Aetna authorizes claim for PoNS Device at out-of-network negotiated price of $18,350, becoming the third payer to join Anthem and United in providing PoNS reimbursement- -Out of Network reimbursement is only the first step towards expanding third-party paid access to PoNS- NEWTOWN, Pa., June 11, 2025 (GLOBE NEWSWIRE) -- Helius Medical Technologies, Inc. (Nasdaq: HSDT) ("Helius" or the "Company"), a neurotech company focused on delivering a novel therapeutic neuromodulation approach for balance and gait def ...

SAN DIEGO and CALGARY, AB, June 11, 2025 /PRNewswire/ -- Oncolytics Biotech® Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapy for oncology, today announced the appointment of Jared Kelly as Chief Executive Officer and a member of its Board of Directors.Mr. Kelly is a successful biotech executive who has proven expertise in transformative deals and corporate strategy. Most recently, he played a central role in orchestrating the sale of Ambrx Biopharma to Johnson ...

-Ends- Enquiries: OXB: Sophia Bolhassan, Head of Investor Relations – T: +44 (0) 1865 509 737 / E: ir@oxb.com Oxford, UK – 11 June 2025: OXB (LSE: OXB) ("the Company"), a global quality and innovation-led cell and gene therapy CDMO, provides an update to coincide with its Annual General Meeting being held at 3pm BST today at Windrush Court, Oxford, UK. OXB has demonstrated strong commercial momentum and made good operational and commercial progress in the year 2025 to date, as it continues to provide viral ...